TfR1-Mediated Delivery of Oligonucleotides to Address Neuromuscular Diseases: Translating Research into Clinical Functional Improvement
Time: 1:00 pm
day: Day 1
Details:
- The FORCE™ platform leverages Transferrin Receptor 1 (TfR1) to enable delivery of therapeutic oligonucleotides to muscle and the CNS
- Discuss preclinical data in models of DM1 and DMD to demonstrate that FORCE™ delivers broad functional improvement in muscle and CNS phenotypes
- The FORCE™ platform achieved POC in the ACHIEVE and DELIVER trials in DM1 and DMD, demonstrating translation of preclinical research into clinical functional improvement
